Global Patent Index - EP 3133070 B1

EP 3133070 B1 20190814 - ELIGLUSTAT (GENZ 112638) AS INHIBITOR OF GLUCOSYLCERAMIDE SYNTHASE FOR USE IN A METHOD OF TREATING FABRY'S OR GAUCHER'S DISEASE, THE METHOD COMPRISING ADJUSTING THE INDIVIDUAL THERAPEUTICAL DOSE TO THE P-450 METABOLISM OF THE PATIENT

Title (en)

ELIGLUSTAT (GENZ 112638) AS INHIBITOR OF GLUCOSYLCERAMIDE SYNTHASE FOR USE IN A METHOD OF TREATING FABRY'S OR GAUCHER'S DISEASE, THE METHOD COMPRISING ADJUSTING THE INDIVIDUAL THERAPEUTICAL DOSE TO THE P-450 METABOLISM OF THE PATIENT

Title (de)

ELIGLUSTAT (GENZ 112638) ALS INHIBITOR DER GLUCOSYLCERAMIDSYNTHASE ZUR VERWENDUNG IN EINEM VERFAHREN ZUR BEHANDLUNG VON FABRY'S ODER GAUCHER'S ERKRANKUNG, WOBEI DAS VERFAHREN DIE ANPASSUNG DER INDIVIDUELLEN THEARAPEUTISCHEN DOSIS AN DEN P-450 STOFFWECHSEL DES PATIENTEN UMFASST

Title (fr)

ELIGLUSTAT (GENZ 112638) EN TANT QUE INHIBITEUR DE LA GLUCOSYLCÉRAMIDE SYNTHASE POUR L'UTILISATION DANS UN PROCÉDÉ POUR TRAITER LA MALADIE DE FABRY OU DE GAUCHER, LE PROCÉDÉ COMPRENANT AJUSTER LA DOSE INDIVIDUELLE AU MÉTABOLISME DU P-450 DU PATIENT

Publication

EP 3133070 B1 20190814 (EN)

Application

EP 16175117 A 20101124

Priority

  • US 26474809 P 20091127
  • EP 16159714 A 20101124
  • EP 14164650 A 20101124
  • EP 10785289 A 20101124
  • US 2010057952 W 20101124

Abstract (en)

[origin: WO2011066352A1] The hemitartrate salt of a compound represented by the following structural formula: (Formula I Hemitartrate), which may be used in pharmaceutical applications, are disclosed. Particular single crystalline forms of the Formula (I) Hemitartrate are characterized by a variety of properties and physical measurements. As well, methods of producing crystalline Formula (I) Hemitartrate, and using it to inhibit glucosylceramide synthase or lowering glycosphingolipid concentrations in subjects to treat a number of diseases, are also discussed. Pharmaceutical compositions are also described.

IPC 8 full level

C07D 405/06 (2006.01); A61K 31/4025 (2006.01); A61K 45/06 (2006.01); A61P 35/00 (2006.01)

CPC (source: CN EP IL KR US)

A61K 31/4025 (2013.01 - CN EP IL KR US); A61K 31/445 (2013.01 - KR); A61K 31/7004 (2013.01 - KR); A61K 45/06 (2013.01 - IL KR US); A61P 3/00 (2018.01 - KR); A61P 35/00 (2018.01 - EP IL); A61P 43/00 (2018.01 - KR); C07D 319/16 (2013.01 - EP IL US); C07D 319/18 (2013.01 - CN); C07D 405/06 (2013.01 - CN EP IL KR US); C07B 2200/13 (2013.01 - CN KR)

Citation (opposition)

Opponent : Accord Healthcare Ltd

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  • MCEACHERN KERRY ANNE ET AL: "A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 91, no. 3, 1 July 2007 (2007-07-01), AMSTERDAM, NL, pages 259 - 267, XP002617211, ISSN: 1096-7192, DOI: 10.1016/J.YMGME.2007.04.001
  • PETERSCHMITT, J.ET AL: "102. Genz-112638, an investigational oral treatment for Gaucher disease type 1: Preliminary Phase 2 clinical trial results", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 96, no. 2, 1 February 2009 (2009-02-01), AMSTERDAM, NL, pages S34, XP025772035, ISSN: 1096-7192, DOI: 10.1016/j.ymgme.2008.11.103
  • PETERSCHMITT, J. LUKINA, E. WATMAN, N. BANIKAZEMI, M. IASTREBNER, M. ROSENBAUM, H. ZIMRAN, A. ARREGUIN, E.A. O'BRI: "73. Preliminary results of a phase II clinical trial of Genz-112638 in patients with type I Gaucher disease", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 93, no. 2, 14 January 2008 (2008-01-14), AMSTERDAM, NL, pages 32 - 33, XP022420706, ISSN: 1096-7192, DOI: 10.1016/j.ymgme.2007.10.085
  • MCEACHERN, K. SIEGEL, C. FUNG, J. CHUANG, W.L. HUTTO, E. SUNG, C. BLANKSTEIN, L. PETERSCHMITT, M.J. GRABOWSKI, G.A: "24 Genz-112638: A novel orally available ceramide-based inhibitor of glucosylceramide synthase for treating Gaucher disease", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 92, no. 4, 20 November 2007 (2007-11-20), AMSTERDAM, NL, pages 17, XP022354774, ISSN: 1096-7192, DOI: 10.1016/j.ymgme.2007.08.029
  • ULRICH M. ZANGER ET AL.: "Cytochrome P450 2D6: Overview and Update on Pharmacology, Genetics", BIOCHEMISTRY , NAUNYN-SCHMIEDEBERG'S ARCHIVES OF PHARMACOLOGY, vol. 369, 2004, pages 23 - 37, XP002555967, DOI: 10.1007/s00210-003-0832-2
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Opponent : Hetero Labs Limited

  • WO 2008150486 A2 20081211 - GENZYME CORP [US], et al
  • WO 03008399 A1 20030130 - GENZYME CORP [US]
  • PETERSCHMITT J ET AL.: "102. Genz-112638, an investigational oral treatment for D1 Gaucher disease type 1: Preliminary Phase 2 clinical trial results", MOLECULAR GENETICS AND METABOLISM, vol. 96, no. 2, 14 January 2009 (2009-01-14), AMSTERDAM, NL, pages S34, XP025772035, DOI: 10.1016/j.ymgme.2008.11.103
  • J.A. SHAYMAN: "Eliglustat tartrate", DRUGS OF THE FUTURE, vol. 35, no. 8, 1 August 2010 (2010-08-01), pages 613 - 620, XP002757375, DOI: 10.1358/dof.2010.035.08.1505566
  • "mechanisms of Disease", 1 January 2008, CAMBRIDGE MEDICINE, article TIMOTHY M COX: "Other stratagems for treating Gaucher's disease", pages: 62 - 63, XP055739869
  • ANONYMOUS: "A Study of Genz-112638 in Patients With Gaucher Disease(ENGAGE) (NCT00891202)", CLINICALTRIALS.GOV ARCHIVE, 3 November 2009 (2009-11-03), XP055378017, Retrieved from the Internet <URL:https://clinicaltrials.gov/archive/NCT00891202/2009_11_03> [retrieved on 20170601]
  • ANONYMOUS: "A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme (NCT00943111)", CLINICALTRIALS.GOV STUDY NCT00943111, 5 November 2009 (2009-11-05), XP055378019, Retrieved from the Internet <URL:https://clinicaltrials.gov/archive/NCT00943111/2009_11_05> [retrieved on 20170601]
  • "Cytochromes P450: Role in the Metabolism and Toxicity of Drugs and other Xenobiotics", 1 January 2008, RSC PUBLISHING, article IOANNIDES COSTAS: "Substrate Binding and Selectivity", pages: 15 - 24, XP055739882
  • "Cytochromes P450: Role in the Metabolism and Toxicity of Drugs and other Xenobiotics", 1 January 2008, RSC PUBLISHER, article IOANNIDES ZANGER: "The CYP2D Subfamily", pages: 255 - 264, XP055739887
  • "The CYP450 Gene Family and Drug Metabolism", BACKGROUND INFORMATION, ROCHE DIAGNOSTICS
  • MCELROY ET AL.: "CYP2D6 Genotyping as an Alternative to Phenotyping for Determination of Metabolic Status in a Clinical Trial Setting", AAPS PHARMSI, vol. 2, no. 4, 2000, XP002251960, DOI: 10.1208/ps020433
  • MCEACHERN, K.A. FUNG, J. KOMARNITSKY, S. SIEGEL, C.S. CHUANG, W.L. HUTTO, E. SHAYMAN, J.A. GRABOWSKI, G.A. AERTS, : "A specific and potent inhibitor of glucosylceramide synthase for substrate inhibition therapy of Gaucher disease", MOLECULAR GENETICS AND METABOLISM, ACADEMIC PRESS, AMSTERDAM, NL, vol. 91, no. 3, 31 May 2007 (2007-05-31), AMSTERDAM, NL, pages 259 - 267, XP022102856, ISSN: 1096-7192, DOI: 10.1016/j.ymgme.2007.04.001
  • BERTILSON L. ET AL.: "Molecular genetics of CYP2D6: clinical relevance with focus on psychotropic drugs", BR J CLIN PHARMACOL., vol. 53, no. 2, February 2002 (2002-02-01), pages 111 - 22, XP055381924, DOI: 10.1046/j.0306-5251.2001.01548.x

Opponent : Teva Pharmaceutical Industries Ltd

  • WO 2008150486 A2 20081211 - GENZYME CORP [US], et al
  • PETERSCHMITT J ET AL.: "102. Genz-112638, an investigational oral treatment for Gaucher disease type 1: Preliminary Phase 2 clinical trial results", MOL GEN & MET, vol. 96, no. 2, 1 February 2009 (2009-02-01), pages S34, XP025772035
  • S TOMLINSON ET AL.: "Mechanism of Disease - An Introduction to Clinical Science. 2nd ed.", 2008, CAMBRIDGE UNIVERSITY PRESS, article "Substrate reduction therapy", pages: 63; 64, XP055704205
  • E LUKINA ET AL.: "Latest Data on Genz-112638, an Investigational Oral Therapy for Type 1 Gaucher Disease:Phase II Clinical Trial Results After 1 Year of Treatment", CLINICAL THERAPEUTICS, vol. 31, no. C, 2009, pages S194 - S195, XP026786231
  • E LUKINA ET AL.: "GENZ-112638, AN INVESTIGATIONAL ORAL THERAPY FOR GAUCHERDISEASE TYPE 1: PHASE 2 CLINICAL TRIAL RESULTS AFTER ONE YEAR OF TREATMENT", HAEMATOLOGICA, vol. 94, no. s2, 2009, pages 41, XP055704206
  • ANONYMOUS: "Study of Genzyme Oral Compound for Gaucher Disease Meets Primary Endpoint", SANOFI PRESS RELEASE, 20 February 2009 (2009-02-20), XP055704207, Retrieved from the Internet <URL:https://www.sanofigenzyme.com/en/about-us/newsroom/archive/2009/2009-02-20-01-45-00>
  • MCEACHEM K ET AL.: "24 Genz-112638: A novel orally available ceramide-based inhibitor of glucosylceramide synthase for treating Gaucher disease", MOL GEN & MET, vol. 92, no. 4, 20 November 2007 (2007-11-20), pages S17, XP022354774
  • ANONYMOUS: "Drug Metabolism and Pharmacokinetics - an overview", EUR PHARM REV, 12 December 2009 (2009-12-12), XP055704210, Retrieved from the Internet <URL:https://www.europeanpharmaceuticalreview.com/article/655/drug-metabolism-and-pharmacokinetics-an-overview/#:~:text=Pharmacodynamics%20(PD)%2C%20which%20studies,include%20PK%20and%20PD%20objectives.&text=Pharmacokinetics%20(PK)%2C%20is%20the,body%20deals%20with%20the%20drug.>
  • BELLE D ET AL.: "Genetic Factors in Drug Metabolism", AMER FAMILY PHYSICIAN, vol. 77, no. 11, 1 June 2008 (2008-06-01), pages 1553 - 1560, XP055704212
  • VAN SCHAIK RON: "Dose Adjustments Based on Pharmacogenetics of CYP450 Enzymes", THE COMMUNICATIONS AND PUBLICATIONS DIVISION (CPD) OF THE IFCC, vol. 19, no. 1, 3 April 2008 (2008-04-03), pages 42 - 47, XP055704214
  • SHARP CF ET AL.: "CYP2D6 genotype and its relationship with metoprolol dose, concentrations and effect in patients with systolic heart failure", THE PHARMACOGENOMICS JOURNAL, vol. 9, no. 3, 1 June 2009 (2009-06-01), pages 175 - 184, XP008134361
  • BOZINA N ET AL.: "Genetic Polymorphism of Metabolic Enzymes P450 (CYP) as a Susceptibility Factor for Drug Response, Toxicity, and Cancer Risk", ARH HG RADA TOKSIKOL, vol. 60, no. 2, 1 June 2009 (2009-06-01), pages 217 - 242, XP055704217
  • ZHOU S ET AL.: "Polymorphism of human cytochrome P450 enzymes and its clinical impact", DRUG MET REV, vol. 41, no. 2, 2009, pages 89 - 295, XP055704222
  • GAEDIGK A ET AL.: "The CYP2D6 Activity Score: Translating Genotype Information into a Qualitative Measure of Phenotype", CLINICAL PHARMACOLOGY AND THERAPEUTICS, vol. 83, no. 2, February 2008 (2008-02-01), pages 234 - 242, XP055704225
  • ANONYMOUS: "A Study of Genz-112638 in Patients With Gaucher Disease Who Have Been Stabilized on Cerezyme (ENCORE)", CLINICALTRIALS.GOV STUDY NCT00943111, 5 November 2009 (2009-11-05), XP055378019, Retrieved from the Internet <URL:https://clinicaltrials.gov/ct2/history/NCT00943111?V_5=View#StudyPageTop>

Designated contracting state (EPC)

AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

Designated extension state (EPC)

BA ME

DOCDB simple family (publication)

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DOCDB simple family (application)

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